Division of Endocrinology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
Department of Pediatrics and Neurology, University of Massachusetts Medical School, Worcester, Massachusetts, USA.
Muscle Nerve. 2020 May;61(5):623-631. doi: 10.1002/mus.26846. Epub 2020 Mar 16.
Recombinant human insulin-like growth factor-1 (rhIGF-1) is a growth factor and has anabolic effects on muscle. We investigated whether rhIGF-1 therapy: 1) improves or preserves muscle function; and 2) improves growth in boys with Duchenne muscular dystrophy (DMD).
In this study we compared prepubescent, ambulatory, glucocorticoid-treated boys with DMD (n = 17) vs controls (glucocorticoid therapy only, n = 21) in a 6-month-long, prospective, randomized, controlled trial of subcutaneous rhIGF-1 therapy. The primary outcome was 6-minute walk distance (6MWD). Secondary outcomes included height velocity (HV), change in height standard deviation score (ΔHtSDS), motor function, cardiopulmonary function, body composition, insulin sensitivity, quality of life, and safety.
Change in 6MWD was similar between groups (rhIGF-1 vs controls [mean ± SD]: 3.4 ± 32.4 vs -5.1 ± 50.2 meters, P = .53). Treated subjects grew more than controls (HV: 6.5 ± 1.7 vs 3.3 ± 1.3 cm/year, P < .0001; 6-month ΔHtSDS: 0.25, P < .0001). Lean mass and insulin sensitivity increased in treated subjects.
In boys with DMD, 6 months of rhIGF-1 therapy did not change motor function, but it improved linear growth.
重组人生长因子-1（rhIGF-1）是一种生长因子，对肌肉具有合成代谢作用。我们研究了 rhIGF-1 治疗是否：1）改善或维持肌肉功能；2）改善杜氏肌营养不良症（DMD）男孩的生长。
在这项研究中，我们将 17 名接受糖皮质激素治疗的青春期前、能行走的 DMD 男孩（n = 17）与对照组（仅接受糖皮质激素治疗，n = 21）进行了比较，这些男孩参与了一项为期 6 个月的前瞻性、随机、对照的 rhIGF-1 皮下治疗试验。主要结局是 6 分钟步行距离（6MWD）。次要结局包括身高速度（HV）、身高标准差评分变化（ΔHtSDS）、运动功能、心肺功能、身体成分、胰岛素敏感性、生活质量和安全性。
两组之间的 6MWD 变化相似（rhIGF-1 组与对照组：3.4 ± 32.4 米与-5.1 ± 50.2 米，P =.53）。治疗组比对照组生长更快（HV：6.5 ± 1.7 厘米/年与 3.3 ± 1.3 厘米/年，P ＜.0001；6 个月 ΔHtSDS：0.25，P ＜.0001）。治疗组的瘦体重和胰岛素敏感性增加。
在 DMD 男孩中，6 个月的 rhIGF-1 治疗并未改变运动功能，但改善了线性生长。
